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BioWorld - Wednesday, December 6, 2023
Breaking News: Topic alerts now available for all BioWorld subscribersBreaking News: Topic alerts now available for all BioWorld subscribersBreaking News: Topic alerts now available for all BioWorld subscribersBreaking News: Topic alerts now available for all BioWorld subscribers
  • An illustration of the connections between muscle fibers and a motor neuron.

    Stathmin-2 is new player in the ALS gene-verse

    A multi-institutional research team has suggested that aberrant TDP-43 processing of the pre-mRNA of a microtubule-associated protein, stathmin-2 (STMN2), may be the primary contributor to amyotrophic lateral sclerosis (ALS).
  • New transthyretin aggregation inhibitor outperforms tolcapone

  • Preveceutical Medical to ramp up preclinical diabetes and obesity dual gene therapy program

  • Oryzon Genomics awarded grant to support preclinical development of ORY-4001 in ALS

  • Longboard’s 5-HT2C superagonist yields positive results in preclinical models of epilepsy

  • Stathmin-2 is new player in the ALS gene-verse

    A multi-institutional research team has suggested that aberrant TDP-43 processing of the pre-mRNA of a microtubule-associated protein, stathmin-2 (STMN2), may be the primary contributor to amyotrophic lateral sclerosis (ALS).
  • New transthyretin aggregation inhibitor outperforms tolcapone

    The misassembly of transthyretin (TTR) into toxic amyloid aggregates leads to a variety of degenerative disorders known as TTR amyloidosis (ATTR). Researchers from the Universitat Autònoma de Barcelona and collaborators recently reported on the design and preclinical characterization of PITB, a TTR-selective kinetic stabilizer with potential for the treatment of ATTR.
  • Preveceutical Medical to ramp up preclinical diabetes and obesity dual gene therapy program

    Preveceutical Medical Inc. is working to accelerate the completion of its preclinical diabetes and obesity dual gene therapy program. The company is pursuing a dual-gene therapy strategy using in vitro validated Smart-siRNA sequences, which when paired with its proprietary bioresponsive lipid-nanoparticulate delivery systems, effectively target the PTPN1 gene.
  • Oryzon Genomics awarded grant to support preclinical development of ORY-4001 in ALS

    Oryzon Genomics SA has been awarded a $498,690 grant by the ALS Association to support the regulatory preclinical development of ORY-4001, a highly selective histone deacetylase 6 (HDAC6) inhibitor, for amyotrophic lateral sclerosis (ALS).
  • Longboard’s 5-HT2C superagonist yields positive results in preclinical models of epilepsy

    Serotonin 5-HT2 receptor agonists have shown efficacy in treating seizure disorders, such as epilepsy. At the American Epilepsy Society meeting this week, Longboard Pharmaceuticals Inc. presented data on their 5-HT2C receptor superagonist LP-352 for the treatment of epilepsy.
  • Holiday notice

    In accordance with the publishing schedule, BioWorld Science will not be published on Wednesday, Dec. 6, 2023.
  • NLS Pharmaceutics enters option agreement with Aexon Labs for dual orexin receptor agonist platform

    NLS Pharmaceutics Ltd. has entered into an exclusive worldwide option agreement with Aexon Labs Inc.
  • GT Biopharma submits IND application for GTB-3650 for CD33+ leukemia

    GT Biopharma Inc. has submitted an IND application to the FDA for the development of GTB-3650 for the treatment of patients with CD33+ leukemia.
  • Seismic Therapeutic closes series B financing to advance immunology pipeline

    Seismic Therapeutic Inc. has closed a $121 million series B financing to support progression of the company’s two lead programs through clinical proof of mechanism.
  • Prelude Therapeutics patents SMARCA2 and SMARCA4 protein degraders for treatment of cancer

    Research at Prelude Therapeutics Inc. has led to the development of SMARCA2 and/or SMARCA4 degradation inducers.
  • Rev-erbA-α agonist ameliorates multiple sclerosis in mice

    Myelinated axons in a rat spinal root.
  • VHL ligands reported in Genentech patent

  • Merck Sharp & Dohme identifies new RSV and MPV inhibitors

  • Mission Therapeutics’ MTX-325 cleared to enter clinic in UK for Parkinson’s disease

    Parkinson's disease illustration showing neurons containing alpha-synuclein
  • Relay Therapeutics describes new PI3Kα inhibitors for cancer

  • Voronoi patent discloses BRAF inhibitors for cancer

  • Simcere’s USP1 inhibitor receives FDA clearance for clinical trials in solid tumors

    3D Rendering of tumor microenvironment
  • Topic alerts now available for all BioWorld subscribers

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Conferences

  • Favorable preclinical profile of investigational gene therapy for TLE reported

    Gene therapy
    Temporal lobe epilepsy (TLE) is the most common form of focal epilepsy characterized by recurrent seizures originating in the hippocampus and with sprouting of mossy fiber axons that contribute to new recurrent synaptic excitability in the dentate gyrus (DG).
  • Brain waves

    Iama Therapeutics’ IAMA-6 shows promise in preclinical mesial temporal lobe epilepsy

    Neurology/psychiatric
    Intracellular chloride levels are crucial for physiological brain development and correct functioning. On the other hand, aberrant expression of chloride importer NKCC1 (basolateral Na-K-Cl symporter, SLC12A2) and exporter KCC2 (K-Cl cotransporter 2, SLC12A5) is involved in neurodevelopmental and...
  • Bifunctional molecule capsid modulator and cGAS agonist achieves complete cure in AAV-HBV mice

    American Association for the Study of Liver Diseases
    LW-231, under development by Shanghai Longwood Biopharmaceuticals Co. Ltd., is a novel bifunctional molecule designed to simultaneously modulate hepatitis B virus (HBV) capsid assembly and activate cGAS-STING pathway. Preclinical data were recently presented.
  • Colorized epithelial cells.

    Stem, immune and nervous cells all forces for tumor progression

    Cancer
    One of the difficulties for preventing the evolution of a tumor is that cancer progression can be promoted by undifferentiated or migrating cells whose states could follow different directions. At the 40th edition of Barcelona Biomed conferences at the Institute for Research in Biomedicine (IRB...
More in Conferences

Today's news in brief

  • Respiratory

  • Dermatologic

  • Cardiovascular

  • Hematologic

  • Nephrology

Patents

  • Idorsia Pharmaceuticals identifies new orexin OX1 and OX2 receptor antagonists

  • Roche divulges EGFR mutant inhibitors for NSCLC

  • Wake Forest University designs doxorubicin codrugs with reduced cardiotoxicity

  • New NLRP3 inflammasome inhibitors reported in Ventus Therapeutics patent

  • Character Biosciences patents new polypeptides for AMD

  • Synrx Therapeutics discovers new POLθ inhibitors for cancer

  • Betta Pharmaceuticals presents new EZH2 inhibitors for cancer

  • Kumquat Biosciences describes new GTPase KRAS inhibitors for cancer

  • Gibson Oncology and Purdue Research Foundation divulge new Topo1 inhibitors and MycG4 ligands

  • Erasca patents new ULK1 inhibitors for cancer

Cancer

  • Microscope with laptop displaying histology image.

    Discovery of dual-target inhibitors of microtubule polymerization and JAK2

    Synthesis and optimization of substituted 2-amino[1,2,4]-triazolopyrimidines and related heterocycles by Wuyi University investigators has led to the identification of compound [I], and its water-soluble phosphate sodium salt prodrug, compound [II].
  • TPT-004, a tryptophan hydroxylase inhibitor with efficacy in MC38 mouse colon carcinoma model

    New compound
  • DCZ-5417 suppresses multiple myeloma progression, study shows

    New compound
  • RNA-binding plus ribosomal proteins block c-Myc in lung cancer

  • China Pharmaceutical University reports discovery of novel EGFR triple-mutation inhibitors

More in Cancer
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Infection

  • Scientist with vial

    Ebviously presents in vitro data on EBV vaccine candidate

    Immune
    Ebviously GmbH, a spin-off from Helmholtz Munich (HMGU), has presented new in vitro data for its Epstein-Barr virus (EBV) vaccine candidate EBV-001. Based on noninfectious virus-like particles (VLPs) derived from EBV, EBV-001 is designed as a highly immunogenic, multiantigen vaccine to prevent...
  • Dual Mcl-1 and Bcl-2 inhibition as promising approach for treating tuberculosis

    Tuberculosis still kills a lot of people worldwide (1.6 million deaths per year). Previous findings demonstrated that induced myeloid leukemia cell differentiation protein Mcl-1 inhibitors reduced the growth of Mycobacterium tuberculosis in human macrophages, but when inhibiting multiple proteins...
  • New dual inhibitors of host proteases TMPRSS2 and CTSL/CTSB as potential anti-SARS-CoV-2 agents

    Coronavirus
    Current antiviral agents for COVID-19 treatment target viral proteins, which are susceptible to mutation during SARS-CoV-2 evolution. A potential strategy to fight emerging drug resistances is the development of compounds targeting host proteins that are indispensable for the viral life cycle.
  • Ethris gains clearance for phase I study of ETH-47 for respiratory viral infections

    Regulatory
  • GHRH-R antagonist attenuates pulmonary dysfunction, heart injury in COVID-19 mice

    Conferences
  • Synthesis and evaluation of new quinoline-piperazine/pyrrolidine compounds against leishmaniasis

  • Zenal Pharmachem divulges new compounds to treat infection

    Patents
More in Infection

Neurology/psychiatric

  • Psychogenics licenses rights to ENT1 inhibitor for neuropsychiatric disorders

    License
    PGI Drug Discovery LLC, the drug discovery arm of Psychogenics Inc., has entered into an exclusive license agreement with F. Hoffmann-La Roche Ltd. to obtain global development, manufacturing and commercialization rights to RO-7117997. Psychogenics intends to evaluate RO-7117997 as a potential...
  • Digital spine concept art

    Nurexone reports new siRNA sequences with potential for spinal cord injury

    RNA
    Nurexone Biologic Inc. has reported results from laboratory tests of its secondary two proprietary sequences, showing promise for the treatment of spinal cord injuries.
  • Cytokinetics describes new bicyclic piperazinones to modulate skeletal muscle contractility

    Patents
    Cytokinetics Inc. has identified bicyclic piperazinones with the ability to modulate skeletal muscle contractility reported to be useful for the treatment of amyotrophic lateral sclerosis, spinal muscular atrophy, myasthenia gravis, chronic obstructive pulmonary disease, stress urinary...
  • Roche divulges new GABRA2 PAMs for medical imaging of neurological disorders

    Diagnostics
    Researchers at F. Hoffmann-La Roche Ltd. and Hoffmann-La Roche Inc. have synthesized radiolabeled compounds acting as GABA(A) receptor subunit α2β2γ1 (GABRA2) positive allosteric modulators acting as positron-emission tomography (PET) and autoradiography imaging agents reported to be useful for the...
  • Brain penetration profile of Nodthera’s NLRP3 inflammasome inhibitor described

    Inflammatory
    Aberrant NLRP3 inflammasome activation is at the root of a wide number of conditions, from heart, gastrointestinal, kidney or liver disorders to neuroinflammatory diseases such as Parkinson’s disease or multiple sclerosis. Researchers from Nodthera Ltd. have reported on NT-0796, an NLRP3...
More in Neurology/psychiatric

Immune

  • New pemphigus vulgaris mouse model better mimics human disorder

  • New RIPK2 scaffolding inhibitor selectively blocks microbe-induced inflammation

  • Abbvie reports preclinical profile of kinase inhibitor ABBV-712

  • Aurinia Therapeutics’ AUR-300 proven useful in systemic sclerosis

  • T-Therapeutics raises $59M in series A for T-cell receptors

  • Anti-CD19 KYV-101 shows promise for autoimmune diseases

  • Emergex to support T cell-based Chikungunya vaccine candidate

  • Guangzhou Fermion Technology presents data on TYK2 inhibitor

  • Scirhom submits CTA for anti-iRhom2 antibody for autoimmune disorders

  • TLR7-targeting antibody shows promise in SLE treatment

Endocrine/metabolic

  • Alpha-galactosidase enzyme

    FDA clearance for first-in-human study of Uniqure’s AMT-191 for Fabry disease

    Regulatory
    Uniqure NV has received FDA clearance of its IND application for AMT-191, the company’s gene therapy candidate for Fabry disease.
  • CSPC Pharmaceutical’s SYH-2053 receives NMPA clearance for clinical studies in China

    Regulatory
  • Translational data on Regeneron’s leptin receptor agonist support further development

    Monoclonal antibody
  • Study links microbiome, vitamin B12, tissue repair in ulcerative colitis

    Microbiome
  • New AMPK-activating oxazole derivative shows antiadipogenic effects

More in Endocrine/metabolic
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Biomarkers

  • miR-92a confers protection against inflammation-driven neurodegeneration

    Neurology/psychiatric
    MicroRNAs (miRNAs) play an important role in modulating neuronal stress responses, but their impact on neuroprotection has been poorly studied. Multiple sclerosis (MS) is the most common inflammatory disease affecting the central nervous system. Researchers have recently attempted to identify...
  • Somatic Ras-MAPK activating variants linked to mesial temporal lobe epilepsy

    Conferences
    Researchers from The Brigham and Women's Hospital presented data from a study that aimed to evaluate the role of somatic variants in drug-resistant mesial temporal lobe epilepsy (MTLE). High-coverage whole-exome sequencing was conducted using DNA samples derived from the hippocampus and paired...
  • Researchers find de novo CAMK2B variant in patient with tetralogy of Fallot

    Conferences
    CAMK2B encodes calcium/calmodulin-dependent protein kinase type II subunit β, a kinase expressed in the brain that has an important role in synaptic plasticity. Genetic variations are associated with neurodevelopmental disorders.
  • Researchers find KAT6B is deleterious variant behind neuroblastoma

    Conferences
  • HE4 and CA-125 as markers of diffuse myocardial fibrosis

    Conferences
  • POLD3 variant linked to immunodeficiency, neurodevelopmental delay

    Conferences
  • Identification of new candidate causative genes of visual impairment in consanguineous families

    Conferences
More in Biomarkers

Gastrointestinal

  • Esperion Therapeutics discovers new ACLY inhibitors

    Patents
    Esperion Therapeutics Inc. has described ATP citrate lyase (ACLY) inhibitors reported to be useful for the treatment of cancer and more.
  • ZHX2 promotes liver recovery after acute liver injury

    Dysfunction in the mitochondria contributes to the development of acute liver injury (ALI). There is emerging evidence indicating the mitochondria is key to maintain liver homeostasis and survival; thus, controlling its functioning is important...
  • Novel hDHODH inhibitor for inflammatory bowel disease described

    Human dihydroorotate dehydrogenase (hDHODH) is a mitochondrial enzyme participating in de novo pyrimidine biosynthesis essential for T- and B-lymphocyte proliferation.
  • Investigators find lactic acid bacteria strain that alleviates IBS in rats

    Irritable bowel syndrome (IBS) is a functional gastrointestinal disorder where visceral hypersensitivity (VH), which causes discomfort and negatively impacts the quality of life, is influenced by stressful events. In recent years, the use of...
  • Lerna Biopharma presents first-in-class GalNAc-siRNA therapeutic for treatment of liver diseases

    Conferences
    Researchers from Lerna Biopharma Pte. Ltd. (formerly Cargene Therapeutics Pte. Ltd.) recently presented the discovery and preclinical evaluation of a first-in-class GalNAc-siRNA therapeutic, CG-LR1, being developed for the treatment of liver...
  • CVI Pharmaceuticals CVI-2742 shows promise for treating NASH

    Conferences
    CVI Pharmaceuticals has presented preclinical data on their thyroid hormone receptor beta (THRB) selective agonist CVI-2742 for the potential treatment of NASH. Selective activation of the THRB contributes to ameliorating the symptoms of...
More in Gastrointestinal

BioWorld Insider Podcast

One-on-one with medical innovators

podcast microphone, sound waves on purple backgroundBreakthrough medicines, billion-dollar deals, spectacular clinical successes and crushing failures all play a part in biopharma’s dynamic story. Developers make scientific advancements with the potential to change everything, only to face regulatory conundrums and ever-fluctuating markets. BioWorld tracks key events in the fast-moving sector every business day. Now, the BioWorld Insider podcast lets you hear directly from the movers and shakers whose collective work is changing how we all live. Join us each week for a new conversation.


Recent episodes:

  • AI: driving drug development from effective to remarkable
  • The struggle is real: The first half of 2023 was an uphill climb
  • The biosimilars challenge to Humira revs up
  • Preventing opioid overdoses with a smart patch
  • Better times ahead for the biopharma sector? Could be, the new numbers say
  • Radiopharmaceuticals: The next big disrupter?
  • Cambrian carves out a new niche as it works to keep people from getting sick
  • Rethinking obesity: Fitness may be more directly linked to health than weight
  • Looking ahead to 2023: CEOs contemplate the new normal
  • $1B+ biopharma deals keep values afloat, even amid muted volume

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